Last update 01 Jul 2024

Calcitriol

Last update 01 Jul 2024

Overview

R&D Status

Clinical Result

Translational Medicine

Overview

Basic Info

Drug Type

Small molecule drug

Synonyms

(1S,3R,5Z,7E)-9,10-secocholesta-5,7,10-triene-1,3,25-triol, (1α,3β,5Z,7E)-9,10-secocholesta-5,7,10(19)-triene-1,3,25-triol, (5Z,7E)-(1S,3R)-9,10-secocholesta-5,7,10(19)-triene-1,3,25-triol

+ [25]

Target

VDR

Mechanism

VDR agonists(Vitamin D receptor agonists)

Therapeutic Areas

Immune System DiseasesEndocrinology and Metabolic DiseaseSkin and Musculoskeletal Diseases+ [3]

Active Indication

Plaque psoriasisChronic Kidney Disease-Mineral and Bone DisorderOsteoporosis, Postmenopausal+ [8]

Inactive Indication

Advanced cancerAdvanced Lung Non-Small Cell CarcinomaAdvanced Pancreatic Adenocarcinoma+ [5]

Originator Organization

Validus Capital Pte Ltd.

Active Organization

Zhejiang Cheng Yi Pharmaceutical Co., Ltd.

SS PharmaceuticalsGalderma Laboratories LP

+ [5]

Inactive Organization

Novacea, Inc.Galderma Research & Development SNC

Hamad Medical Corp.

Drug Highest PhaseApproved

First Approval Date

US (17 Aug 1978),

Hyperparathyroidism, SecondaryKidney Failure, Chronic

Regulation-

Structure

Molecular FormulaC27H44O3

InChIKeyGMRQFYUYWCNGIN-NKMMMXOESA-N

CAS Registry32222-06-3

177

Clinical Trials associated with Calcitriol

CTR20240267 / RecruitingNot Applicable

骨化三醇胶丸在中国健康受试者中空腹和餐后状态下的随机、开放、两序列、两周期交叉设计生物等效性试验

[Translation] A randomized, open-label, two-sequence, two-period crossover bioequivalence study of calcitriol softgels in Chinese healthy subjects under fasting and fed conditions

主要研究目的研究空腹和餐后状态下单次口服骨化三醇胶丸（规格：0.25μg，山东简道制药有限公司）与参比制剂（规格：0.25μg，罗盖全®；持证商：Roche Pharma (Schweiz) AG）后骨化三醇在中国健康受试者体内的药代动力学（PK）特征，评价空腹和餐后状态下口服两种制剂的生物等效性。次要研究目的评价中国健康受试者空腹和餐后状态下，单次口服受试制剂骨化三醇胶丸和参比制剂骨化三醇胶丸后的安全性。

[Translation]

Main study objectives To study the pharmacokinetic (PK) characteristics of calcitriol in healthy Chinese subjects after a single oral administration of calcitriol capsules (specification: 0.25μg, Shandong Jiandao Pharmaceutical Co., Ltd.) and reference preparation (specification: 0.25μg, Rogaquan®; licensee: Roche Pharma (Schweiz) AG) in fasting and fed states, and to evaluate the bioequivalence of the two preparations in fasting and fed states. Secondary study objectives To evaluate the safety of the test preparation calcitriol capsules and reference preparation calcitriol capsules in healthy Chinese subjects after a single oral administration in fasting and fed states.

Start Date20 May 2024

Sponsor / Collaborator

Shandong Jiandao Pharmaceutical Co., Ltd.

ChiCTR2400083304 / RecruitingNot ApplicableIIT

A Single-Center, Open-Label, Randomized, Single-Dose, Two-Period, Two-Sequence, Crossover Trial to Assess the Bioequivalence of Test Product Calcitriol Capsules (Strength: 0.5 mcg) and Reference Product Calcitriol Capsules (CALCITRIOL, Strength: 0.5 mcg) in Healthy Adult Participants under Fed Conditions

Start Date10 Apr 2024

Sponsor / Collaborator-

CTR20240248 / RecruitingNot Applicable

骨化三醇胶丸在中国健康受试者中的一项随机、开放、空腹和餐后单次给药、两制剂、两序列、两周期交叉设计的生物等效性试验

[Translation] A randomized, open-label, single-dose, fasting and fed, two-formulation, two-sequence, two-period crossover bioequivalence study of calcitriol softgels in healthy Chinese subjects

主要研究目的研究空腹和餐后状态下单次口服骨化三醇胶丸（规格：0.25μg，大连美创药业有限公司）与参比制剂（规格：0.25μg，罗盖全®；持证商：Roche Pharma (Schweiz) AG）后骨化三醇在中国健康受试者体内的药代动力学（PK）特征，评价空腹和餐后状态下口服两种制剂的生物等效性。次要研究目的评价中国健康受试者空腹和餐后状态下，单次口服受试制剂骨化三醇胶丸和参比制剂骨化三醇胶丸后的安全性。

[Translation]

Main study objectives To study the pharmacokinetic (PK) characteristics of calcitriol in healthy Chinese subjects after a single oral administration of calcitriol capsules (specification: 0.25μg, Dalian Meichuang Pharmaceutical Co., Ltd.) and reference preparation (specification: 0.25μg, Rogaquan®; licensee: Roche Pharma (Schweiz) AG) in fasting and fed states, and to evaluate the bioequivalence of the two preparations in fasting and fed states. Secondary study objectives To evaluate the safety of the test preparation calcitriol capsules and reference preparation calcitriol capsules in healthy Chinese subjects after a single oral administration in fasting and fed states.

Start Date26 Mar 2024

Sponsor / Collaborator

Dalian Meichuang Pharmacy Co., Ltd.

100 Clinical Results associated with Calcitriol

100 Translational Medicine associated with Calcitriol

100 Patents (Medical) associated with Calcitriol

11,409

Literatures (Medical) associated with Calcitriol

01 Mar 2024·Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics

Calcitriol ameliorates motor deficits and prolongs survival of Chrne-deficient mouse, a model for congenital myasthenic syndrome, by inducing Rspo2

Article

Author: Inoue, Taro ; Ohkawara, Bisei ; Kanbara, Shunsuke ; Nakashima, Hiroaki ; Takeda, Jun-Ichi ; Tomita, Hiroyuki ; Koshimizu, Hiroyuki ; Ito, Mikako ; Ogi, Tomoo ; Ohno, Kinji ; Zhang, Shaochuan ; Miyasaka, Yuki ; Imagama, Shiro ; Nishiwaki, Hiroshi ; Ishiguro, Naoki ; Ohno, Tamio ; Masuda, Akio

Signal transduction at the neuromuscular junction (NMJ) is compromised in a diverse array of diseases including congenital myasthenic syndromes (CMS). Germline mutations in CHRNE encoding the acetylcholine receptor (AChR) ε subunit are the most common cause of CMS. An active form of vitamin D, calcitriol, binds to vitamin D receptor (VDR) and regulates gene expressions. We found that calcitriol enhanced MuSK phosphorylation, AChR clustering, and myotube twitching in co-cultured C2C12 myotubes and NSC34 motor neurons. RNA-seq analysis of co-cultured cells showed that calcitriol increased the expressions of Rspo2, Rapsn, and Dusp6. ChIP-seq of VDR revealed that VDR binds to a region approximately 15 kbp upstream to Rspo2. Biallelic deletion of the VDR-binding site of Rspo2 by CRISPR/Cas9 in C2C12 myoblasts/myotubes nullified the calcitriol-mediated induction of Rspo2 expression and MuSK phosphorylation. We generated Chrne knockout (Chrne KO) mouse by CRISPR/Cas9. Intraperitoneal administration of calcitriol markedly increased the number of AChR clusters, as well as the area, the intensity, and the number of synaptophysin-positive synaptic vesicles, in Chrne KO mice. In addition, calcitriol ameliorated motor deficits and prolonged survival of Chrne KO mice. In the skeletal muscle, calcitriol increased the gene expressions of Rspo2, Rapsn, and Dusp6. We propose that calcitriol is a potential therapeutic agent for CMS and other diseases with defective neuromuscular signal transmission.

14 Feb 2024·The British journal of nutrition

Efficacy of vitamin D₂ in maintaining serum total vitamin D concentrations and bone mineralisation in adult dogs fed a plant-based (vegan) diet in a 3-month randomised trial

Article

Author: Dewey, Cate ; Khosa, Deep ; Verbrugghe, Adronie ; Abood, Sarah K ; Dodd, Sarah A S ; Adolphe, Jennifer

Abstract:

Dogs are considered omnivores based on their evolution consuming diets including animal tissue. Few feeding trials evaluating the nutritional suitability of exclusively plant-based (vegan) diets in dogs have been published, and the efficacy of vitamin D2 in maintaining canine serum vitamin D levels has not been clearly determined. A blinded dietary trial included sixty-one healthy desexed adult dogs: thirty-one fed an experimental extruded vegan diet (PLANT) and thirty fed a commercial extruded meat-based diet (MEAT) for 3 months. Dogs were screened via veterinary examination and routine laboratory analyses prior to enrolment, at baseline and exit timepoints. Body composition was measured by dual-energy X-ray absorptiometry and blood was collected for vitamin D profiling. All dogs maintained health parameters, body weight and composition throughout the study. Dogs maintained on PLANT demonstrated a significant reduction in platelet count, creatinine, blood urea nitrogen and cholesterol, though values remained within normal reference ranges. Dogs fed PLANT also demonstrated a shift from vitamin D3 to vitamin D2 metabolites, though total vitamin D analogue levels were unchanged, with the exception of 24,25-dihydroxyvitamin D. Bone mineral content and density did not differ from baseline values. Health status was maintained in dogs fed PLANT and vitamin D2 appeared efficacious in maintaining serum total vitamin D concentrations and bone mineralisation. Findings support the hypothesis that PLANT was comparable to MEAT for maintenance of healthy adult dogs for at least 3 months and identified areas where further research is warranted to elucidate the potential risks and benefits of plant-based (vegan) diets.

01 Feb 2024·Saudi pharmaceutical journal : SPJ : the official publication of the Saudi Pharmaceutical Society

Biological evaluation of combinations of tyrosine kinase inhibitors with Inecalcitol as novel treatments for human chronic myeloid leukemia

Article

Author: Saleh, Maysaa M ; Abuarqoub, Duaa A ; Alyoussef Alkrad, Jamal ; Al-Ali, Luma ; Al-Ani, Raad J ; Najdawi, Manal M ; Naser, Abdallah Y ; Abbas, Manal M ; Hammad, Alaa M ; Abu-Irmaileh, Bashaer

Background:

The use of tyrosine kinase inhibitors (TKIs) as a treatment for chronic myeloid leukemia (CML) has improved the natural history of the disease and increased the duration of survival. Tyrosine kinase inhibitors represent the success of target therapies that work on molecular targets, although some patients still have therapy failure. Vitamin D has antiproliferative, pro-apoptotic, and anti-angiogenic effects on cells, therefore it can be considered as a potential cancer preventative and treatment agent. Inecalcitol (TX-522) is the 14-epi-analogue of Calcitriol (1,25(OH)₂-vitamin D3), and inhibits cancer cell proliferation more effectively than Calcitriol. This study was conducted to evaluate the antiproliferative and synergistic effects of the anticancer drugs Imatinib and Dasatinib in combinations with Inecalcitol on human chronic myeloid leukemia K-562 cells.

Method:

The growth inhibitory activities of Inecalcitol, Imatinib, Dasatinib, and different combinations of one of the two drugs (Imatinib and Dasatinib) with Inecalcitol, were determined in vitro using MTT assay against K-562 cell line.

Results:

Inecalcitol, Imatinib, and Dasatinib showed potent antiproliferative activities against K-562 cells with GI₅₀ values of 5.6 µM, 0.327 µM, and 0.446 nM, respectively. Combinations of Imatinib or Dasatinib with different concentrations of Inecalcitol increased significantly the antiproliferative activities and potencies of both drugs (****p < 0.0001), with optimal GI₅₀ values of 580 pM (Imatinib) and 0.51 pM (Dasatinib). Furthermore, the combination treatments showed synergistic interaction between the antileukemic drugs and Inecalcitol, with combination indices (CI) < 1.

Conclusion:

The study demonstrated that the human chronic myeloid leukemia K-562 cells were subjected to a synergistic growth inhibitory impact when antileukemic drugs (Imatinib or Dasatinib) were combined with Inecalcitol, therefore, it is recommended that these combinations be viewed as promising novel antileukemic medications and used in place of individual medications with lower dosages and negligible side effects in the treatment of CML.

News (Medical) associated with Calcitriol

13 May 2024

·www.globenewswire.com

Entera Bio to Present Phase 1 Data from First-in-Class, PTH(1-34) Peptide Tablets (EB612) for the Treatment of Hypoparathyroidism at ENDO 2024

JERUSALEM, May 13, 2024 (GLOBE NEWSWIRE) -- Entera Bio Ltd. (NASDAQ: ENTX), (“Entera” or the “Company”) a leader in the development of orally delivered peptides and small therapeutic proteins, today announced that it has been selected to present data for its investigational program EB612 at the Endocrine Society ENDO 2024 Annual Meeting which will be held on June 1-4, 2024 in Boston, Massachusetts. The EB612 program is being developed by Entera to provide the first oral PTH(1-34) tablet peptide replacement therapy for patients suffering from hypoparathyroidism. Hypoparathyroidism is characterized by deficient PTH production, hypocalcemia and hyperphosphatemia. Standard treatments include several daily administrations of high dose oral calcium supplements and calcitriol (or analogs), which are often associated with ectopic calcification, including nephrocalcinosis and renal failure. Late stage investigational PTH replacement treatments include TransCon PTH (palopegteriparatide) by Ascendis Pharma A/S and eneboparatide (AZP-3601) by Amolyt Pharma (pending acquisition by AstraZeneca). Both these modalities require patients to administer injections every day. The data from this Phase 1 study include PK and early PD results from the application of a new generation of Entera’s N-Tab™ technology platform with an unmodified PTH(1-34) peptide. One of the objectives of this study was to test the new platform’s ability to reduce the frequency of daily administration of EB612. Entera previously published positive Phase 2a study results in 19 hypoparathyroid patients using a QID regimen (Fraser, JBMR 2021). Abstract Title: Phase 1 Study Results Of EB612, A First-in-Class Oral PTH(1-34) Analog For The Treatment Of Hypoparathyroidism Presentation Number: SAT-758 Presentation Type: Late-Breaking Poster Presentations: Day 1 Session: Poster Session P106 Session Date/Time: Saturday, June 1, 2024, 12:15 pm – 1:45 pm The abstract can be found on the ENDO 2024 website: https://www.abstractsonline.com/pp8/#!/11016/presentation/9825 About Entera Bio Entera is a clinical stage company focused on developing oral peptide or protein replacement therapies for significant unmet medical needs where an oral tablet form holds the potential to transform the standard of care. The Company leverages on a disruptive and proprietary technology platform (N-Tab™) and its pipeline includes five differentiated, first-in-class oral peptide programs, expected to enter the clinic (Phase 1 to Phase 3) by 2025. The Company’s most advanced product candidate, EB613 (oral PTH(1-34)), is being developed as the first oral, osteoanabolic (bone building) once-daily tablet treatment for post-menopausal women with low BMD and high-risk osteoporosis, with no prior fracture. A placebo controlled, dose ranging Phase 2 study of EB613 tablets (n= 161) met primary (PD/bone turnover biomarker) and secondary endpoints (BMD). Entera is preparing to initiate a Phase 3 registrational study for EB613 pursuant to the FDA’s qualification of a quantitative BMD endpoint which is expected to occur by January 2025. The EB612 program is being developed as the first oral PTH(1-34) tablet peptide replacement therapy for hypoparathyroidism. Entera is also developing the first oral oxyntomodulin, a dual targeted GLP1/glucagon peptide, in tablet form for the treatment of obesity; and first oral GLP-2 peptide tablet as an injection-free alternative for patients suffering from rare malabsorption conditions such as short bowel syndrome in collaboration with OPKO Health. For more information on Entera Bio, visit www.enterabio.com or follow us on LinkedIn, Twitter, Facebook, Instagram. Cautionary Statement Regarding Forward Looking Statements Various statements in this press release are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. All statements (other than statements of historical facts) in this press release regarding our prospects, plans, financial position, business strategy and expected financial and operational results may constitute forward-looking statements. Words such as, but not limited to, “anticipate,” “believe,” “can,” “could,” “expect,” “estimate,” “design,” “goal,” “intend,” “may,” “might,” “objective,” “plan,” “predict,” “project,” “target,” “likely,” “should,” “will,” and “would,” or the negative of these terms and similar expressions or words, identify forward-looking statements. Forward-looking statements are based upon current expectations that involve risks, changes in circumstances, assumptions and uncertainties. Forward-looking statements should not be read as a guarantee of future performance or results and may not be accurate indications of when such performance or results will be achieved. Important factors that could cause actual results to differ materially from those reflected in Entera’s forward-looking statements include, among others: changes in the interpretation of clinical data; results of our clinical trials; the FDA’s interpretation and review of our results from and analysis of our clinical trials; unexpected changes in our ongoing and planned preclinical development and clinical trials, the timing of and our ability to make regulatory filings and obtain and maintain regulatory approvals for our product candidates; the potential disruption and delay of manufacturing supply chains; loss of available workforce resources, either by Entera or its collaboration and laboratory partners; impacts to research and development or clinical activities that Entera may be contractually obligated to provide; overall regulatory timelines; the size and growth of the potential markets for our product candidates; the scope, progress and costs of developing Entera’s product candidates; Entera’s reliance on third parties to conduct its clinical trials; Entera’s expectations regarding licensing, business transactions and strategic collaborations; Entera’s operation as a development stage company with limited operating history; Entera’s ability to continue as a going concern absent access to sources of liquidity; Entera’s ability to obtain and maintain regulatory approval for any of its product candidates; Entera’s ability to comply with Nasdaq’s minimum listing standards and other matters related to compliance with the requirements of being a public company in the United States; Entera’s intellectual property position and its ability to protect its intellectual property; and other factors that are described in the “Cautionary Statements Regarding Forward-Looking Statements,” “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations” sections of Entera’s most recent Annual Report on Form 10-K filed with the SEC, as well as the company’s subsequently filed Quarterly Reports on Form 10-Q and Current Reports on Form 8-K. There can be no assurance that the actual results or developments anticipated by Entera will be realized or, even if substantially realized, that they will have the expected consequences to, or effects on, Entera. Therefore, no assurance can be given that the outcomes stated or implied in such forward-looking statements and estimates will be achieved. Entera cautions investors not to rely on the forward-looking statements Entera makes in this press release. The information in this press release is provided only as of the date of this press release, and Entera undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise, except to the extent required by law.

Phase 2Phase 1Clinical ResultAcquisitionPhase 3

14 Mar 2024

·www.prnewswire.com

Psoriasis in America - Insights into Psoriasis Patient Demographics, Quality of Life, Information-seeking Behaviors, HCP Engagement and Treatment Experiences

DUBLIN, March 14, 2024 /PRNewswire/ -- The "Psoriasis in America 2023: Understanding the Psoriasis Patient Experience" report has been added to ResearchAndMarkets.com's offering. Approximately 7.5 million people in the United States have psoriasis. This Psoriasis in America report provides a comprehensive look at life for those living with psoriasis. Using detailed quantitative data, it gives a full picture of the psoriasis patient experience - from symptoms to treatment to HCP relationships to quality of life - so you can make informed, strategic decisions, identify opportunities, and understand potential challenges. The report offers an in-depth look at the psoriasis patient experience. This large-scale, patient-reported data leverages vital quantitative insights essential to understanding key touchpoints in the psoriasis patient journey, including condition management, HCP engagement, quality of life, treatment experience and satisfaction, and much more. This report lifts the curtain on life with psoriasis, giving stakeholders an actionable look at the behaviors, attitudes, perceptions, needs, and experiences of people living with the condition. Data can be used to inform strategic decisions, including product communications, competitive assessments, concept development, landscape analyses, patient journey overlays, and forecasting inputs. This report also addresses important questions that stakeholders may not even know to ask while offering valuable insights into must-have patient-reported data points not available anywhere else. Add-on custom data analysis opportunities are also available for an additional cost - please see the report following purchase for more information. The analyst reaches millions of people through its portfolio of 45+ condition-specific online health communities - including PlaquePsoriasis.com - to provide information, connection, and support to patients and caregivers in the U.S. This report includes a deep-dive into: Psoriasis patient demographics Age, gender, ethnicity, marital status, children, employment status, income, location, insurance, and other health conditions Impact of psoriasis on quality of life Severity, impact on quality of life, symptoms experienced, and what patients wish others better understood Information-seeking behaviors Information sources used, plus topics of interest HCP engagement Primary HCP seen for condition, satisfaction with HCP, and discussion about brands aware of/not used Psoriasis treatment awareness and experiences Aided awareness of specific treatments, treatment experience, satisfaction with current treatments, perceived control with current treatment plan, and interest/participation in clinical trials Key questions answered in this report: To what extent do people say psoriasis impacts their overall quality of life? What's the average number of flares in the past year? What do patients wish others understood about psoriasis? What percentage of patients see a specialist for psoriasis treatment? What treatments have the highest aided brand awareness among patients? What percentage of patients use a biologic? How many patients feel their psoriasis is well-controlled on their current treatment plan? What percentage of patients are likely to switch or add new treatments in the next six months? What medications have patients discussed with their HCP? What are the top online resources people with psoriasis use to find information? What kinds of content and information do patients search for? What percentage of patients search for information on treatment options? Methodology Psoriasis in America consists of: A 20-minute online quantitative survey, covering demographics, comorbidities, quality of life/impact, HCP interactions, as well as treatment awareness, experiences, and discussions Qualitative patient insights from open-ended responses Additional details: Fielded: January 16, 2023 to April 14, 2023 Convenience sample of 490 respondents diagnosed with psoriasis Respondents are age 18+, living in the U.S., and are recruited from proprietary online health communities and recruiting partners Key Topics Covered: Respondent Demographics Research Highlights Condition Status and Quality of Life Severity and Exacerbations Symptoms Experienced Impact on Quality of Life Qualitative Patient Insights Information-Seeking Behaviors Resources Used to Manage Health Types of Content/Information Sought Treatment Awareness and Experiences Primary HCP Seen for Psoriasis Care Aided Brand Awareness of Treatments Treatment Usage Condition Control on Current Treatment and Clinical Trial Interest Treatment Discussions with HCP Appendix with All Data Charts and Distributions Brands and treatments mentioned in this report include: Topical corticosteroid (such as hydrocortisone) BRYHALIT (halobetasol propionate) lotion DesOwen (desonide) SERNIVOT (betamethasone dipropionate) SORILUXT Foam (calcipotriene) TACLONEX (calcipotriene and betamethasone) TAZORAC (tazarotene) VECTICAL (calcitriol) VTAMA (tapinarof) ZITHRANOLT-RR (anthralin) ZORYVET (roflumilast) Acitretin (SORIATANE) ARAVA (leflunomide) Azathioprine (such as IMURAN, AZASAN) Cyclosporine (such as Sandimmune, NEORAL, RESTASIS) Methotrexate (eg, Rheumatrex), also sometimes given as an injection OTEZLA (apremilast) PLAQUENIL (hydroxychloroquine) RINVOQ (upadacitinib) SOTYKTUT (deucravacitinib) Sulfasalazine (Azulfidine) XELJANZ/XELJANZ XR (tofacitinib citrate) AMJEVITAT (adalimumab-atto) Hide until launched in US AVSOLA (infliximab-axxq) CIMZIA (certolizumab) COSENTYX (secukinumab) CYLTEZO (adalimumab-adbm) Hide until launched in US ENBREL (etanercept) ERELZIT (etanercept-szzs) Hide until launched in US HULIO (adalimumab-fkjp) Hide until launched in US HUMIRA (adalimumab) HYRIMOZ (adalimumab-adaz) Hide until launched in US ILUMYAT (tildrakizumab-asmn) INFLECTRA (infliximab-dyyb) IXIFIT (infliximab-qbtx) Hide until launched in US ORENCIA (abatacept) REMICADE (infliximab) RENFLEXIST (infliximab-abda) SIMPONI (golimumab) SIMPONI ARIA (golimumab for infusion) SILIQT (brodalumab) SKYRIZI (risankizumab-rzaa) STELARA (ustekinumab) TALTZ (ixekizumab) TREMFYA (guselkumab) For more information about this report visit About ResearchAndMarkets.com ResearchAndMarkets.com is the world's leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends. Media Contact: Research and Markets Laura Wood, Senior Manager [email protected] For E.S.T Office Hours Call +1-917-300-0470 For U.S./CAN Toll Free Call +1-800-526-8630 For GMT Office Hours Call +353-1-416-8900 U.S. Fax: 646-607-1907 Fax (outside U.S.): +353-1-481-1716 Logo: SOURCE Research and Markets

02 Nov 2023

·www.biospace.com

New Clinical Data on OPKO Health’s RAYALDEE® (ER Calcifediol) Presented at Kidney Week 2023

MIAMI, Nov. 02, 2023 (GLOBE NEWSWIRE) -- OPKO Health, Inc. (NASDAQ: OPK) presented late-breaking clinical data on RAYALDEE® extended-release calcifediol (ERC) at the American Society of Nephrology (ASN) Kidney Week in Philadelphia today. These data, presented in a poster titled “Control of Secondary Hyperparathyroidism with Extended-release Calcifediol is Associated with Slower CKD Progression” (#TH-PO1152), indicate that early, sustained and effective treatment of secondary hyperparathyroidism (SHPT) with RAYALDEE is associated with significantly slower progression of chronic kidney disease (CKD) in pre-dialysis patients. The poster is available on OPKO’s website here. Progressive changes in estimated glomerular filtration rate (eGFR) were examined post-hoc in 166 patients with vitamin D insufficiency, SHPT and stage 3 or 4 CKD during one year of treatment with RAYALDEE in pivotal trials. The average eGFR decline was 7.7% per year but differed significantly and proportionately with the achieved duration of intact parathyroid hormone (iPTH) control, defined as ≤100 pg/mL, being greatest (16.4%) in patients who never achieved control and least (1.7%) in those achieving consistent control. The number of patients experiencing an increase in eGFR by the end of treatment rose from 3.6% to 10.8% as the duration of iPTH control increased. Treatment with RAYALDEE was not associated with clinically meaningful increases in serum calcium or phosphorus. “Secondary hyperparathyroidism is associated with more rapid CKD progression and earlier dialysis, but mitigation of disease progression by effective control of SHPT has not been previously examined,” stated Charles W. Bishop, Ph.D., CEO of OPKO Health’s Renal Division. “RAYALDEE is a safe and highly effective treatment for SHPT in patients with stage 3 or 4 CKD. The new data presented today clearly highlight the possibility that raising serum 25-hydroxyvitamin D to a sufficiently high level to achieve consistent iPTH control with RAYALDEE would improve outcomes in CKD patients.” About RAYALDEE® RAYALDEE is an extended-release (ER) oral formulation of calcifediol, a prohormone of calcitriol, the active form of vitamin D3. The product is the first and only medicine approved by the U.S. Food and Drug Administration for raising serum total 25D and lowering blood levels of intact parathyroid hormone (iPTH). RAYALDEE is approved to treat SHPT in adults with stage 3 or 4 CKD and vitamin D insufficiency in the U.S. and in 11 European countries. Slowing CKD progression with RAYALDEE treatment is not currently an approved indication. About OPKO Health, Inc. OPKO is a multinational biopharmaceutical and diagnostics company that seeks to establish industry-leading positions in large, rapidly growing markets by leveraging its discovery, development, and commercialization expertise and novel and proprietary technologies. For more information, visit . Cautionary Statement Regarding Forward-Looking Statements This press release contains "forward-looking statements," as that term is defined under the Private Securities Litigation Reform Act of 1995 (PSLRA), which statements may be identified by words such as "expects," "plans," "projects," "will," “could,” "may," "anticipates," "believes," "should," "intends," "estimates," and other words of similar meaning, including statements regarding the market for RAYALDEE, and our strategies or prospects and expectations about RAYALDEE, the therapeutic benefits, safety pro effectiveness of RAYALDEE or whether early initiation of SHPT treatment with RAYALDEE would delay disease progression. Many factors could cause our actual activities or results to differ materially from the activities and results anticipated in forward-looking statements. These factors include those described in our Annual Reports on Form 10-K filed and to be filed with the Securities and Exchange Commission and in our other filings with the Securities and Exchange Commission, as well as the risks that the accuracy and effectiveness of the data may not be reproducible or indicative of future results and that currently available over-the-counter and prescription products, as well as products under development by others, may prove to be as or more effective than our products for the indications being studied. In addition, forward-looking statements may also be adversely affected by general market factors, competitive product development, product availability, federal and state regulations and legislation, the regulatory process for new products and indications, manufacturing issues that may arise, patent positions and litigation, among other factors. The forward-looking statements contained in this press release speak only as of the date the statements were made, and we do not undertake any obligation to update forward- looking statements. We intend that all forward-looking statements be subject to the safe- harbor provisions of the PSLRA. Contacts: LHA Investor Relations Yvonne Briggs, 310-691-7100 ybriggs@lhai.com or Bruce Voss, 310-691-7100 bvoss@lhai.com

Clinical ResultDrug Approval

100 Deals associated with Calcitriol

External Link

KEGG	Wiki	ATC	Drug Bank
D00129	Calcitriol	D05AX03 A11CC04	DB00136

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Plaque psoriasis	US	Galderma Laboratories LP	23 Jan 2009
Chronic Kidney Disease-Mineral and Bone Disorder	CN	Roche Pharma AG	17 Sep 1999
Osteoporosis, Postmenopausal	CN	Roche Pharma AG	17 Sep 1999
Bone Diseases, Metabolic	US	SS Pharmaceuticals	20 Nov 1998
Osteoporosis	JP	Chugai Pharmaceutical Co., Ltd.	11 Sep 1989
Vitamin D Deficiency	JP	Chugai Pharmaceutical Co., Ltd.	05 Nov 1985
Hyperparathyroidism, Secondary	US	SS Pharmaceuticals	17 Aug 1978
Kidney Failure, Chronic	US	SS Pharmaceuticals	17 Aug 1978

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Diabetic Nephropathies	Phase 3	QA	Hamad Medical Corp.	10 May 2017
Castration-Resistant Prostatic Cancer	Phase 3	US	Novacea, Inc.	01 Jan 2006
Castration-Resistant Prostatic Cancer	Phase 3	CA	Novacea, Inc.	01 Jan 2006
Castration-Resistant Prostatic Cancer	Phase 3	CZ	Novacea, Inc.	01 Jan 2006
Castration-Resistant Prostatic Cancer	Phase 3	DE	Novacea, Inc.	01 Jan 2006
Castration-Resistant Prostatic Cancer	Phase 3	HU	Novacea, Inc.	01 Jan 2006
Castration-Resistant Prostatic Cancer	Phase 3	ME	Novacea, Inc.	01 Jan 2006
Castration-Resistant Prostatic Cancer	Phase 3	PR	Novacea, Inc.	01 Jan 2006
Castration-Resistant Prostatic Cancer	Phase 3	RO	Novacea, Inc.	01 Jan 2006
Castration-Resistant Prostatic Cancer	Phase 3	SK	Novacea, Inc.	01 Jan 2006

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03469271 (CTgov)	Phase 4	Neoplasms \| Pneumonia	63	Inspiratory isometric resistance training (Training and Placebo)	dptsddgjvo(hhhzfieovq) = sriyflmqri azgqcyllkj (ixebogeqxy, pighkovoyf - itznosikhk) View more	-	26 Sep 2023
				Threshold Inspiratory Muscle Trainer (IMT)+1,25 (OH)2 D3 (Sham Training and Vitamin D3 Metabolite)	dptsddgjvo(hhhzfieovq) = kigegvuxlj azgqcyllkj (ixebogeqxy, yrqjnzwmfx - qumazvmsks) View more
EASD2022	Not Applicable	Diabetes Mellitus, Type 2 \| Chronic Kidney Diseases intact parathyroid hormone (iPTH)	45	Calcitriol 0.5 mcg once daily	cxfendwroc(cxlbfooqaj) = scqtqixbuq idkbhijqzx (rfdqibnqce, 12.1) View more	Negative	22 Sep 2022
				Placebo	cxfendwroc(cxlbfooqaj) = rtrkbsgmwc idkbhijqzx (rfdqibnqce, 14.74) View more
NCT00763555 (CTgov)	Phase 2	Plaque psoriasis	88	CD 2027 (Calcitriol 3 mcg/g Spray)	oiepbglunb(kqhkmljpgd) = xptfsnwxlt qbtutiungx (grqmhcdtju, drhxcvpztp - quztyxkuhg) View more	-	23 Aug 2022
				Calcitriol Vehicle (Calcitriol Vehicle)	oiepbglunb(kqhkmljpgd) = srnsblkyxh qbtutiungx (grqmhcdtju, qpzwkxctep - gwtzaaimjn) View more
NCT04286529 (CTgov)	Phase 1	Prediabetic State	17	Calcitriol capsules (Men-Calcitriol)	xuofqyxbsi(vjuysuhzql) = dmvkeeojup qufpeqfkel (ogdmfmibpt, rlvfkrdkwo - jbkkkfmsgr) View more	-	18 Apr 2022
				Calcitriol capsules (Premenopausal Women-Calcitriol)	xuofqyxbsi(vjuysuhzql) = vgrbzgsuet qufpeqfkel (ogdmfmibpt, tiaavvkxau - kuklejmcie) View more
NCT02962102 (ACTIVATE-AKI, CTgov)	Phase 2	Critical Illness \| Acute Kidney Injury	150	Calcifediol (Calcifediol)	nkqskkssqs(zjnpwahbjf) = mxiunhbdjn bzvvvgxfge (wracckzmcb, zusfcugksu - zqctmxsupu) View more	-	03 Feb 2022
				Calcitriol (Calcitriol)	nkqskkssqs(zjnpwahbjf) = ndbpipsbwj bzvvvgxfge (wracckzmcb, gxuwznmsmw - uasjxknnpj) View more
VIKI Study (ASN2021)	Not Applicable	vitamin K \| VKDPs \| vitamin 25(OH) D ... View more	387	Oral Calcitriol Use	pwtvfujddh(mtcfiwlrvk) = htakbaimxv rcijsnxhnm (hwegnigmhe )	Positive	27 Oct 2021
				No Oral Calcitriol Use	pwtvfujddh(mtcfiwlrvk) = ilikknpgvh rcijsnxhnm (hwegnigmhe )
NCT02061384 (RA-2, CTgov)	Phase 2	Male Sterility Due to Y-Chromosome Deletions \| Klinefelter Syndrome \| Oligospermia	20	13-cis retinoic acid (13-cis Retinoic Acid)	mjxrpjdzrl(fxwkdduach) = nfwyqtqzng upcmqgunzh (xtlptqvozk, otptelfmse - iudfskwcdz) View more	-	30 Jul 2021
				Calcitriol+13-cis retinoic acid (Calcitriol 0.25 mcg)	mjxrpjdzrl(fxwkdduach) = zisrgeqssv upcmqgunzh (xtlptqvozk, fawmpjhhgp - utrqjqbvab)
VIKI (ERA2021)	Not Applicable	Hemodialysis complication Vitamin K levels \| VKDPs \| vitamin 25(OH)D ... View more	387	Oral Calcitriol	qszefjxhja(rsemcslvbu) = ewygsdnqak nyahsptute (mwinubpjhi )	Positive	29 May 2021
				calcitriol	qszefjxhja(rsemcslvbu) = ufjnoqxqzv nyahsptute (mwinubpjhi )
MO1020 (ERA2021)	Not Applicable	-	26	Calcitriol	gdlzqoqxgp(lreiyvdvsl) = lfcbujijpp yfauurafbe (qdhifcidsa ) View more	Positive	29 May 2021
				Alfacalcidol	gdlzqoqxgp(lreiyvdvsl) = bjyrqcugib yfauurafbe (qdhifcidsa ) View more
NCT03261336 (CTgov)	Phase 2	Castration-Resistant Prostatic Cancer	1	Calcitriol+Ketoconazole+Hydrocortisone	bldwjmfjyy(bnrqebitsx) = hwskglqrow ybigdohppi (codxddaznq, sgombmyuyz - eyxammdfap) View more	-	08 Feb 2021

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